Patient Advocacy Takes the Stage at MassBio’s Inaugural Summit

The first annual Massachusetts Biotechnology Council (MassBio) Patient Advocacy Summit was held on Monday, November 10, 2014 at the Genzyme Center in Cambridge. The sold-out Summit, titled “Live & Breathe: Building a Patient-Centered Biotech,” featured a full day of panel discussions and case studies that gave disease advocacy organizations and biotechnology companies the opportunity to openly discuss some of the most pertinent issues they face today.

Topics included the importance of taking a truly patient-focused philosophy, patient advocacy’s role in the regulatory process, the gray areas associated with company-patient interactions like clinical trials, and issues of access to treatments. The event included top support from sponsors Genzyme, Millennium Pharmaceuticals and Shire, and also Biogen Idec, OvaScience, and Vertex Pharmaceuticals.

MassBio’s impassioned President and CEO Bob Coughlin opened the program with some heartfelt remarks revealing to the crowd why the subject matter of Patient Advocacy was so personal to him and why he had been such a big supporter of the Summit during its planning phases. Coughlin explained he is the parent of a son who suffers with cystic fibrosis (CF) and that he was in attendance also as a parent and advocate. He cited the Cystic Fibrosis Foundation as being one disease organization that has done a great job of building trust and listening to patient communities.

Nancy Roach, Founder of Fight Colorectal Cancer, has been an advocate since 1996 when her mother-in-law was diagnosed, and she herself is also a cancer survivor. She delivered the opening keynote address of the Summit, describing her patient-centric approach from a patient perspective. She argued clinical trials that aren’t going to fundamentally change treatment are “borderline unethical” and she covered many contentious issues like the incredible waste of resources on repetitive activities, the lack of transparency with clinical trial data, unnecessary delays due to bureaucracy, and recruitment and retention failures.

The first panel was moderated by David Meeker, CEO of Genzyme. Meeker’s message to the industry was to bring patients to scientists on the cutting edge of discovery, as he said these scientists represent hope for patients. Composed of Robert Forrester, President and CEO of Verastem; Steve Perrin, CEO of ALS Therapy Development Institute; and Michelle Dipp, CEO and Co-Founder of OvaScience, the panel discussed the broader importance of patient advocacy.

“We spoke with patient advocates even before we started our company,” Dipp said, emphasizing the importance her company places on patient centricity. She also introduced the concept of a Chief Patient Officer position. “My passion for patients is very strong and it’s at front of my mind, but I still need a person whose sole focus is patient advocacy,” she said.

Robert Forrester echoed this by reciting a famous quote from George Merck, founder of pharmaceutical company Merck & Co: “We try never to forget that medicine is for the people. It is not for the profits. The profits follow, and if we have remembered that, they have never failed to appear. The better we have remembered it, the larger they have been.” Steve Perrin added his two cents. “Extending life matters a lot, but extending quality of life is what matters the most and you learn this in that patient conversation,” he said.

Many of the diseases featured at the Summit were referred to as “orphan diseases,” or diseases that affect such a small percentage of the population that they are not deemed financially viable for drug development by pharmaceutical companies. Cystic fibrosis is an example of one of these uncomfortable, painful conditions that affects the lungs, pancreas, liver and intestines. Biotechnology companies like Vertex Pharmaceuticals, have shown that orphan diseases can still be extremely profitable even when focusing on patient populations that are few in number, which was a point of discussion at the event. Their latest breakthrough CF drug, Kalydeco, has a monthly retail price of $30,725 and would not be available to most patients if it were not for the assistance of advocacy groups.

Another panel discussion focused on grey areas – the tricky areas where miscommunication or unclear expectations can exist in company-patient interaction. Sue Kahn, Executive Director of the National Tay-Sachs & Allied Diseases Association discussed her experience. “What patient advocates want to see is collaboration and transparency on how progress can be made for treatments,” she said. Another panelist, John Richert, VP of MS franchise for Biogen Idec, said, “The patient organizations can serve as conveners to help answer the important questions. Companies can’t do this.”

The Summit’s afternoon panels included discussions on the regulatory approval process and how patient voices can help, as well as a discussion on issues of access to treatments, from pricing decisions and insurance questions to the difficult decisions around compassionate use.

Overall, the Summit certainly achieved its goal of bringing together patient advocates and industry leaders to examine ways to more fully integrate the patient voice into the work they do. In fact, there are already discussions happening about next year’s 2nd Annual Patient Advocacy Summit and, if the inaugural event was any indication, we look forward to attending.

Robert Schultz has an MBA in Information Systems from University of Massachusetts-Boston and a BS in International Business from Northeastern University, where he served as Business Manager for the university’s largest student publication, The Northeastern News. Schultz is an experienced healthcare technology startup enthusiast who was involved with the patient monitoring company Aware Engineering through the MassBio MassCONNECT program.

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